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Friday, August 26, 2016

A Tough Week

We made it through a week of appointments at Cincinnati Children's Hospital. Bobby's heart and lungs are stable right now. His overall muscle strength is mostly stable with decline in time to rise from the floor and climbing stairs. At home he no longer attempts to get up off the floor unless he crawls to furniture first and pushes up on it. He was able to get up from a criss cross position on the floor using his hands on the floor and bottom up in the air and actually stood up without pushing on his legs. Dr Wong explained that he had a significant increase in the amount of time it took him to get up that way since February, so that is likely why he uses furniture all the time now. It is simply easier and more efficient for him to do so now. Bobby also walked 450 meters for his 6 minute walk test. He was out of breathe and about to collapse, but was determined to do his best as he knew I needed to know that number in order to determine our next steps for possible drug trials.
We had a rough start to our appointments with the IV line needed for the heart MRI and then 2 rounds of eye drops. Bobby was a bit over the top. So he and I had a long talk about the drug trial we hope will start soon. I explained that the drug would be by infusion and he would have to get an IV stick once a week. I also explained that he could not react and cry and scream like he had for the MRI iv stick. We discussed whether he wanted to take part in the drug trial or not. He is now 10 and he needs to be a part of this decision. He said that he still wanted to do the drug trial. He said that it would fix his legs so he had to do it. I was honest with him and discussed that the exon skipping drugs could slow, not cure or fix his muscle weakness, and that he would most likely need several different drugs or treatments in order to eventually find the cure for his DMD.
Today was our last day of appointments. We talked on the way to the hospital again about how he needed to show me that he would be able to do what he would need to do to take part in the drug trial. Today was the glucose intolerance test. He had to have another IV/port put in and this time have it in for 2 hours. He also had to drink the glucose drink and he absolutely hates sicky sweet things. He said that he could do it, but he did ask if I could talk to him while they stuck the needle in to distract him.
He was nervous as they began to get things out. The nurse explained that she had to go check on another patient in 4 minutes. She said that if he could be calm she could have him done and started with his drink early or if he wanted she would come back after the other patient. Bobby wanted to be able to get done as fast as possible so he could go see his friends so he chose for her to go ahead. He started to be a tad squirmy but the calmed down with talk about the new cat game he had played and actually watched as she put the needle in. No crying, no screaming, no trying to pull away. He the chugged the drink in about a minute and a half. He was perfect for the whole test as he built with Lego in between blood draws. He was pretty proud of himself and let me know that he could handle to drug trial.
I asked about the status of the ESSENCE drug trial while with Dr Wong. She stated that the IRB is ongoing and under review. The budget has already been set and so they are just waiting on the review and approval from Sarepta to be able to begin recruitment. The hope is that they will begin recruiting in October. I am hoping that this timeline while come to fruition and that the trial will actually start. Personally I am not convinced Sarepta will begin the trial unless they get a yes from the FDA for Eteplirsen. Since we are moving over the 90 day since the last FDA delay, I am frustrated and angry. We have already been waiting 2 years for this to start and there just continues to be delays.
All of our trips for the 6 month and 12 month appointments leave me tired. The 7 hour drive to get here and then several days of numerous appointments followed up with the 7 hour drive home is draining. This trip has taken more of a toll. There is a high emotional toll when one has to discuss with their 10 year old what could happen without a drug trial. To discuss the loss of skills that will happen without intervention causes tears to well in both of our eyes. Sometimes it seems too much to process.
Bobby continues to have a positive outlook. He saw a couple of cool power chairs and said "those are fast. I would like one, but my legs are going to get better and my muscular dystrophy will be gone so I won't need one!" That is definitely my hope to and with so many other positive trials that are upcoming, I think that a cure is close. To get there though, we need the FDA to get off of their butts and do what they are tasked to do. They need to stop stonewalling every Duchenne Drug that comes before them and use the flexibility afforded to them under FDASIA. If they can't and continue to discount patient input and data that shows clinical benefit with no negative side effects then they need to be replaced with people who are willing to handle the task at hand and stop further delays. Delays=Decline. Decline eventually leads to death. We have lost enough of our DMD community due to delay after delay. FDA the time is now to answer and the answer you should be giving is "Yes!"